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Phase 2 Pilot Trial of Ruxolitinib Combined With Danazol for Patients With Primary Myelofibrosis (MF), Post Essential Thrombocythemia-Myelofibrosis (Post ET) and Post Polycythemia Vera Myelofibrosis (PV MF) Suffering From Anemia

Phase 2
18 Years
Open (Enrolling)
Anemia, Primary Myelofibrosis, Secondary Myelofibrosis

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Trial Information

Phase 2 Pilot Trial of Ruxolitinib Combined With Danazol for Patients With Primary Myelofibrosis (MF), Post Essential Thrombocythemia-Myelofibrosis (Post ET) and Post Polycythemia Vera Myelofibrosis (PV MF) Suffering From Anemia


I. To evaluate the efficacy (best overall response) of ruxolitinib (ruxolitinib phosphate)
and danazol in patients with myelofibrosis suffering from anemia.


I. To evaluate the overall survival of patients with myelofibrosis suffering from anemia
initiating ruxolitinib and danazol.

II. To evaluate the adverse event profile of ruxolitinib and danazol in patients with
myelofibrosis suffering from anemia.


I. To evaluate quality of life (QOL) and patient-reported symptoms using the
Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) and European Organization for
Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 (QLQ-C30)
with ruxolitinib and danazol for patients with myelofibrosis suffering from anemia.


Patients receive ruxolitinib phosphate orally (PO) twice daily (BID) and danazol PO thrice
daily (TID) on days 1-56. Treatment repeats every 56 days for up to 6 courses in the absence
of disease progression or unacceptable toxicity.

Inclusion Criteria:

- Histological confirmation of primary myelofibrosis (MF), post polycythemia vera (PV)
or post essential thrombocythemia (ET) myelofibrosis (intermediate 1, intermediate II
or high risk) requiring medical therapy

- Anemia is required for trial entry (defined as hemoglobin < 10g/dL or transfusion

- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 at study

- Absolute neutrophil count (ANC) >= 1000 x 10^3

- Platelet count >= 50,000 × 10^3

- Serum creatinine =< 1.5 x the upper limit of normal (ULN)

- Total bilirubin =< 1.5 x ULN; if total bilirubin is > 1.5 x ULN, a direct bilirubin
should be performed and must be < 1.5mg/dL

- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =< 3 x ULN;
higher values (i.e., =< 5 x ULN) are allowed if clinically compatible with hepatic
extramedullary hematopoiesis

- Life expectancy of >= 6 months

- Patient able to provide voluntary written informed consent to participate

- Willing to comply with scheduled visits, treatment plans, laboratory assessments, and
other study-related procedures

- Negative pregnancy test done =< 7 days prior to registration, for women of
childbearing potential only

Exclusion Criteria:

- Any chemotherapy (e.g., hydroxyurea), immunomodulatory drug therapy (e.g.,
thalidomide, interferon-alpha), immunosuppressive therapy, corticosteroids > 10
mg/day prednisone or equivalent, or growth factor treatment (e.g., erythropoietin),
hormones (e.g., androgens, danazol) =< 14 days prior to registration

- Major surgery =< 28 days or radiation =< 6 months prior to registration

- Co-morbid systemic illnesses or other severe concurrent disease which, in the
judgment of the investigator, would make the patient inappropriate for entry into
this study or interfere significantly with the proper assessment of safety and
toxicity of the prescribed regimens

- Active acute infection requiring antibiotics

- Uncontrolled congestive heart failure (New York Heart Association classification 3 or
4), angina, myocardial infarction, cerebrovascular accident, coronary/peripheral
artery bypass, graft surgery, transient ischemic attack, or pulmonary embolism within
3 months prior to registration

- Participation in any study of an investigational agent (drug, biologic, device) =< 30
days, unless during non-treatment phase

- Any of the following because this study involves an agent that has known genotoxic,
mutagenic and teratogenic effects:

- Pregnant women

- Nursing women

- Men or women of childbearing potential who are unwilling to employ adequate

- Known human immunodeficiency virus or acquired immunodeficiency syndrome-related

- Clinically active hepatitis B or C

- Active malignancy other than MF, except adequately treated basal cell carcinoma and
squamous cell carcinoma of the skin, cervical carcinoma in situ or other malignancies
that have been stable and off therapy for 5 years

- Patient currently taking simvastatin, or lovastatin at a dose greater than 10 mg/day

- Men with prostate specific antigen (PSA) > 4 ng/ml or with uncontrolled benign
prostatic hypertrophy

- Receiving any medications or substances that are strong or moderate inhibitors of
cytochrome P450, family 3, subfamily A, polypeptide 4 (CYP3A4); use of the following
strong or moderate inhibitors are prohibited =< 7 days prior to registration

- Strong inhibitors of CYP3A4:

- Indinavir (Crixivan)

- Nelfinavir (Viracept)

- Atazanavir (Reyataz)

- Clarithromycin (Biaxin, Biaxin XL)

- Itraconazole (Sporanox)

- Ketoconazole (Nizoral)

- Nefazodone (Serzone)

- Saquinavir (Fortovase, Invirase)

- Telithromycin (Ketek)

- Moderate inhibitors of CYP3A4

- Erythromycin (Erythrocin, E.E.S., Ery-Tab, Eryc, EryPed, PCE)

- Fluconazole (Diflucan)

- Grapefruit juice

- Verapamil (Calan, Calan SR, Covera-HS, Isoptin SR, Verelan)

- Verelan PM

- Diltiazem (Cardizem, Cardizem CD, Cardizem LA, Cardizem SR, Cartia XT,
Dilacor XR, Diltia XT, Taztia XT, Tiazac)

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Supportive Care

Outcome Measure:

Best overall response rate as determined by International Working Group criteria

Outcome Description:

The proportion of successes will be estimated by the number of successes (defined as complete response [CR], partial response [PR], or clinical improvement [CI]) divided by the total number of evaluable patients. Confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.

Outcome Time Frame:

Up to 2 years

Safety Issue:


Principal Investigator

Ruben Mesa

Investigator Role:

Principal Investigator

Investigator Affiliation:

Mayo Clinic in Arizona


United States: Food and Drug Administration

Study ID:




Start Date:

April 2013

Completion Date:

Related Keywords:

  • Anemia
  • Primary Myelofibrosis
  • Secondary Myelofibrosis
  • Primary Myelofibrosis
  • Anemia
  • Thrombocythemia, Essential



Mayo Clinic in Arizona Scottsdale, Arizona  85259-5404